CA3146364A1 - Therapie genique en une etape pour dystrophie musculaire de duchenne par remplacement de gene et anti-inflammation - Google Patents

Therapie genique en une etape pour dystrophie musculaire de duchenne par remplacement de gene et anti-inflammation Download PDF

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Publication number
CA3146364A1
CA3146364A1 CA3146364A CA3146364A CA3146364A1 CA 3146364 A1 CA3146364 A1 CA 3146364A1 CA 3146364 A CA3146364 A CA 3146364A CA 3146364 A CA3146364 A CA 3146364A CA 3146364 A1 CA3146364 A1 CA 3146364A1
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gene
seq
promoter
sequence
muscle
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Pending
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CA3146364A
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Bing Wang
Freddie FU
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University of Pittsburgh
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University of Pittsburgh
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Dans un mode de réalisation, l'invention concerne un véhicule génique à deux cassettes comprenant des cassettes pour l'expression à la fois d'un gène de la mini-dystrophine et de NF-?B/p65-shARN dans un tissu musculaire cardiaque et un tissu musculaire squelettique, qui est un vecteur viral adéno-associé, le gène de mini-dystrophine étant lié de manière fonctionnelle à une construction comprenant un premier promoteur spécifique du muscle et un activateur de créatine kinase musculaire modifié et le gène NF-?B/p65-shARN étant sous la commande d'un second promoteur. L'invention concerne également des compositions pharmaceutiques comprenant de tels véhicules de gènes et un procédé d'amélioration de la dystrophie musculaire de Duchenne (DMD) utilisant de tels véhicules d'administration de gènes et des compositions pharmaceutiques.
CA3146364A 2019-02-02 2020-02-03 Therapie genique en une etape pour dystrophie musculaire de duchenne par remplacement de gene et anti-inflammation Pending CA3146364A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800484P 2019-02-02 2019-02-02
US62/800,484 2019-02-02
PCT/US2020/016410 WO2020160542A1 (fr) 2019-02-02 2020-02-03 Thérapie génique en une étape pour dystrophie musculaire de duchenne par remplacement de gène et anti-inflammation

Publications (1)

Publication Number Publication Date
CA3146364A1 true CA3146364A1 (fr) 2020-08-06

Family

ID=71841302

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3146364A Pending CA3146364A1 (fr) 2019-02-02 2020-02-03 Therapie genique en une etape pour dystrophie musculaire de duchenne par remplacement de gene et anti-inflammation

Country Status (8)

Country Link
US (1) US20220136005A1 (fr)
EP (1) EP3918081A4 (fr)
JP (1) JP2022523038A (fr)
CN (1) CN113710809A (fr)
AU (1) AU2020216501A1 (fr)
CA (1) CA3146364A1 (fr)
IL (1) IL285129A (fr)
WO (1) WO2020160542A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023248251A1 (fr) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur Vecteur aav optimisé pour la thérapie génique de la dystrophie musculaire

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7266458B2 (en) * 2000-03-06 2007-09-04 Bioseek, Inc. BioMAP analysis

Also Published As

Publication number Publication date
EP3918081A4 (fr) 2022-11-02
JP2022523038A (ja) 2022-04-21
US20220136005A1 (en) 2022-05-05
AU2020216501A1 (en) 2021-09-16
CN113710809A (zh) 2021-11-26
WO2020160542A1 (fr) 2020-08-06
IL285129A (en) 2021-09-30
EP3918081A1 (fr) 2021-12-08

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